HOPE-3 Trial Overview - The HOPE-3 Phase 3 trial met both the primary endpoint (PUL v20) and the key secondary cardiac endpoint (LVEF), achieving statistical significance with p=003 and p=004 respectively[27] - Statistical significance was achieved in all type 1 error controlled secondary endpoints[27] - The trial involved 106 patients randomized 1:1 to Deramiocel or placebo[13, 14] - The study population consisted of late-ambulatory patients with DMD and 10MWR > 10 seconds[15] Safety Profile - In the Placebo group, 827% (43/52) experienced any Treatment Emergent Adverse Events (TEAEs), while in the Deramiocel group, 943% (50/53) experienced any TEAEs[18] - TEAEs related to the Investigational Product (IP) or administration procedure were reported in 365% (19/52) of the placebo group and 830% (44/53) of the Deramiocel group[18] - Serious TEAEs occurred in 96% (5/52) of the placebo group and 19% (1/53) of the Deramiocel group[18] Deramiocel Potential - Deramiocel is a potential first-in-class therapy designed to treat DMD skeletal and cardiomyopathy[27] - The company plans to submit a response to the Complete Response Letter incorporating HOPE-3 data, following prior alignment with the FDA[27]